Kenneth C. Fan, MD, MBA, of Retina Consultants of Texas, highlighted findings from the STARLIGHT study at the American Academy of Ophthalmology 2025 annual meeting, held October 18-10, in Orlando, Florida. He focused on the use of MCO-010 optogenetic therapy in the treatment of Stargardt disease, an inherited retinal dystrophy for which no approved therapies currently exist. The study represents an important step in the exploration of gene-based and novel therapeutic interventions for inherited retinal diseases (IRDs). He described STARLIGHT as a Phase 2 open-label study designed primarily to evaluate safety and tolerability, while also assessing efficacy signals related to visual function. He noted, “Safety and tolerability is one of the most important things in a Phase 2 trial.” Throughout the study, no incidents of retinitis or vasculitis were observed. A small number of participants developed transient vitreous cells, but these findings resolved fully by the end of the study period. Although safety remained the principal end point, efficacy outcomes were notable—particularly among participants with a macular phenotype of Stargardt disease, which Fan referred to as “more typical Stargardt disease.” Among these patients, meaningful improvements in visual acuity were documented at 48 weeks, both with and without the use of a wearable magnifier. Fan highlighted that “the most pronounced improvements of visual acuity were noted in 3 of the patients with a macular phenotype, wherein significant meaningful gains in vision were noted at 48 weeks with and without the wearable magnifier.” Given the absence of any approved treatment for Stargardt disease, these preliminary outcomes are encouraging. As Fan said, “The difficult thing with seeing patients with Stargardt disease is that there's no approved therapy… and so we're happy to see people be able to get any sort of treatment to stabilize or potentially improve the vision.” He suggested that the response observed in the macular phenotype subgroup provides valuable insight for future trial design, noting that it could “be a great guidance for the registrational Phase 3 study in the future to select those patients so that we can maximize visual improvement in patients.” Beyond the data itself, Fan placed the findings in a broader clinical and research context. The progress seen in Stargardt disease mirrors the growing momentum in the development of therapies for inherited retinal diseases (IRDs), including retinitis pigmentosa and other rare genetic disorders. “There’s just really been a significant growth of interest in IRDs and drug development for IRDs in the past couple of years,” he noted, underscoring the rapid expansion of the field. “I’m just hopeful for the future of treatment for patients with inherited retinal disease, not just Stargardt disease, but retinitis pigmentosa and other more rare conditions,” Fan added
